Cazzola M. Myelodysplastic Syndromes. N Engl J Med. 2020 Oct 1. 383 (14):1358-1374. [Medline].
Besa EC. Myelodysplastic syndromes (refractory anemia). A perspective of the biologic, clinical, and therapeutic issues. Med Clin North Am. 1992 May. 76(3):599-617. [Medline].
Dao KT. Myelodysplastic Syndromes: Updates and Nuances. Med Clin North Am. 2017 Mar. 101 (2):333-350. [Medline].
Goldberg H, Lusk E, Moore J, Nowell PC, Besa EC. Survey of exposure to genotoxic agents in primary myelodysplastic syndrome: correlation with chromosome patterns and data on patients without hematological disease. Cancer Res. 1990 Nov 1. 50(21):6876-81. [Medline]. [Full Text].
Liew E, Owen C. Familial myelodysplastic syndromes: a review of the literature. Haematologica. 2011 Oct. 96 (10):1536-42. [Medline]. [Full Text].
Sperling AS, Gibson CJ, Ebert BL. The genetics of myelodysplastic syndrome: from clonal haematopoiesis to secondary leukaemia. Nat Rev Cancer. 2017 Jan. 17 (1):5-19. [Medline]. [Full Text].
Kristinsson SY, Bjorkholm M, Hultcrantz M, et al. Chronic immune stimulation might act as a trigger for the development of acute myeloid leukemia or myelodysplastic syndromes. J Clin Oncol. 2011 Jul 20. 29(21):2897-903. [Medline]. [Full Text].
Bejar R, Steensma DP. Recent developments in myelodysplastic syndromes. Blood. 2014 Oct 30. 124 (18):2793-803. [Medline]. [Full Text].
Key Statistics for Myelodysplastic Syndromes. American Cancer Society. Available at http://www.cancer.org/cancer/myelodysplasticsyndrome/detailedguide/myelodysplastic-syndromes-key-statistics. January 22, 2018; Accessed: January 26, 2021.
Rollison DE, Hayat M, Smith M, et al. First report of national estimates of the incidence of myelodysplastic syndromes and chronic myeloproliferative disorders from the U.S. SEER program [abstract 247]. Blood. 2006. 108:77a. [Full Text].
Rollison DE, Howlader N, Smith MT, et al. Epidemiology of myelodysplastic syndromes and chronic myeloproliferative disorders in the United States, 2001-2004, using data from the NAACCR and SEER programs. Blood. 2008 Jul 1. 112(1):45-52. [Medline]. [Full Text].
Ma X. Epidemiology of myelodysplastic syndromes. Am J Med. 2012 Jul. 125 (7 Suppl):S2-5. [Medline].
Ma X, Does M, Raza A, Mayne ST. Myelodysplastic syndromes: incidence and survival in the United States. Cancer. 2007 Apr 15. 109(8):1536-42. [Medline]. [Full Text].
Roman E, Smith A, Appleton S, Crouch S, Kelly R, Kinsey S, et al. Myeloid malignancies in the real-world: Occurrence, progression and survival in the UK's population-based Haematological Malignancy Research Network 2004-15. Cancer Epidemiol. 2016 Apr 15. [Medline]. [Full Text].
Bennett JM, Catovsky D, Daniel MT, et al. Proposed revised criteria for the classification of acute myeloid leukemia. A report of the French-American-British Cooperative Group. Ann Intern Med. 1985 Oct. 103(4):620-5. [Medline].
Harris NL, Jaffe ES, Diebold J, Flandrin G, Muller-Hermelink HK, Vardiman J, et al. World Health Organization classification of neoplastic diseases of the hematopoietic and lymphoid tissues: report of the Clinical Advisory Committee meeting-Airlie House, Virginia, November 1997. J Clin Oncol. 1999 Dec. 17(12):3835-49. [Medline]. [Full Text].
Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19. 127 (20):2391-405. [Medline]. [Full Text].
Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997 Mar 15. 89(6):2079-88. [Medline]. [Full Text].
Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20. 120 (12):2454-65. [Medline]. [Full Text].
Voso MT, Fenu S, Latagliata R, Buccisano F, Piciocchi A, Aloe-Spiriti MA, et al. Revised International Prognostic Scoring System (IPSS) predicts survival and leukemic evolution of myelodysplastic syndromes significantly better than IPSS and WHO Prognostic Scoring System: validation by the Gruppo Romano Mielodisplasie Italian Regional Database. J Clin Oncol. 2013 Jul 20. 31 (21):2671-7. [Medline].
[Guideline] NCCN Clinical Practice Guidelines in Oncology. Myelodysplastic Syndromes. National Comprehensive Cancer Network. Available at http://www.nccn.org/professionals/physician_gls/pdf/mds.pdf. Version 3.2021 — January 15, 2021; Accessed: January 26, 2021.
Adema V, Bejar R. What lies beyond del(5q) in myelodysplastic syndrome?. Haematologica. 2013 Dec. 98(12):1819-21. [Medline].
Molldrem JJ, Leifer E, Bahceci E, et al. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6. 137(3):156-63. [Medline]. [Full Text].
Myelodysplastic Syndromes Treatment (PDQ®)–Health Professional Version. National Cancer Institute. Available at http://www.cancer.gov/cancertopics/pdq/treatment/myelodysplastic/HealthProfessional/page1#Reference1.9. February 1, 2019; Accessed: January 26, 2021.
Angelucci E, Urru SA, Pilo F, Piperno A. Myelodysplastic Syndromes and Iron Chelation Therapy. Mediterr J Hematol Infect Dis. 2017. 9 (1):e2017021. [Medline]. [Full Text].
[Guideline] Bennett JM, MDS Foundation's Working Group on Transfusional Iron Overload. Consensus statement on iron overload in myelodysplastic syndromes. Am J Hematol. 2008 Nov. 83 (11):858-61. [Medline]. [Full Text].
Kim GYG, Burns J, Freyer CW, Hamilton KW, Frey NV, Gill SI, et al. Risk of Invasive Fungal Infections in Patients with High-Risk MDS and AML Receiving Hypomethylating Agents. Am J Hematol. 2020 Apr 3. [Medline].
Musto P, Lanza F, Balleari E, et al. Darbepoetin alpha for the treatment of anaemia in low-intermediate risk myelodysplastic syndromes. Br J Haematol. 2005 Jan. 128(2):204-9. [Medline].
Jadersten M, Montgomery SM, Dybedal I, Porwit-MacDonald A, Hellstrom-Lindberg E. Long-term outcome of treatment of anemia in MDS with erythropoietin and G-CSF. Blood. 2005 Aug 1. 106(3):803-11. [Medline]. [Full Text].
Clark RE, Jacobs A, Lush CJ, Smith SA. Effect of 13-cis-retinoic acid on survival of patients with myelodysplastic syndrome. Lancet. 1987 Apr 4. 1 (8536):763-5. [Medline].
Crisà E, Foli C, Passera R, Darbesio A, Garvey KB, Boccadoro M, et al. Long-term follow-up of myelodysplastic syndrome patients with moderate/severe anaemia receiving human recombinant erythropoietin + 13-cis-retinoic acid and dihydroxylated vitamin D3: independent positive impact of erythroid response on survival. Br J Haematol. 2012 Jul. 158 (1):99-107. [Medline].
List A, Dewald G, Bennett J, Giagounidis A, Raza A, Feldman E, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5. 355 (14):1456-65. [Medline]. [Full Text].
List A, Kurtin S, Roe DJ, et al. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005 Feb 10. 352(6):549-57. [Medline]. [Full Text].
Saunthararajah Y. Key clinical observations after 5-azacytidine and decitabine treatment of myelodysplastic syndromes suggest practical solutions for better outcomes. Hematology Am Soc Hematol Educ Program. 2013. 2013:511-21. [Medline].
Silverman LR, Demakos EP, Peterson BL, et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002 May 15. 20(10):2429-40. [Medline]. [Full Text].
FDA approves oral combination of decitabine and cedazuridine for myelodysplastic syndromes. U.S. Food & Drug Administration. Available at https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-oral-combination-decitabine-and-cedazuridine-myelodysplastic-syndromes. July 7, 2020; Accessed: Jluy 8, 2020.
FDA Approves New Therapy for Myelodysplastic Syndromes (MDS) That Can Be Taken at Home. U.S. Food & Drug Administration. Available at https://www.fda.gov/news-events/press-announcements/fda-approves-new-therapy-myelodysplastic-syndromes-mds-can-be-taken-home. July 7, 2020; Accessed: July 8, 2020.
Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar. 10(3):223-32. [Medline].
Bejar R, Lord A, Stevenson K, Bar-Natan M, Pérez-Ladaga A, Zaneveld J, et al. TET2 mutations predict response to hypomethylating agents in myelodysplastic syndrome patients. Blood. 2014 Oct 23. 124 (17):2705-12. [Medline]. [Full Text].
Parikh AR, Olnes MJ, Barrett AJ. Immunomodulatory treatment of myelodysplastic syndromes: antithymocyte globulin, cyclosporine, and alemtuzumab. Semin Hematol. 2012 Oct. 49 (4):304-11. [Medline]. [Full Text].
Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. N Engl J Med. 2020 Jan 9. 382 (2):140-151. [Medline].
Scalzulli E, Pepe S, Colafigli G, Breccia M. Therapeutic strategies in low and high-risk MDS: What does the future have to offer?. Blood Rev. 2020 Mar 31. 100689. [Medline].
Platzbecker U. Who benefits from allogeneic transplantation for myelodysplastic syndromes?: new insights. Hematology Am Soc Hematol Educ Program. 2013. 2013:522-8. [Medline].
Arslan S, Nakamura R. Decision Analysis of Transplantation for Patients with Myelodysplasia: "Who Should We Transplant Today?". Curr Hematol Malig Rep. 2020 Mar 28. [Medline].
Sandhu KS, Brunstein C, DeFor T, Bejanyan N, Arora M, Warlick E, et al. Umbilical Cord Blood Transplantation Outcomes in Acute Myelogenous Leukemia/Myelodysplastic Syndrome Patients Aged ≥70 Years. Biol Blood Marrow Transplant. 2015 Sep 28. [Medline].
Shaffer BC, Ahn KW, Hu ZH, Nishihori T, Malone AK, et al. Scoring System Prognostic of Outcome in Patients Undergoing Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Syndrome. J Clin Oncol. 2016 Apr 4. [Medline].
Lindsley RC, Saber W, Mar BG, Redd R, Wang T, Haagenson MD, et al. Prognostic Mutations in Myelodysplastic Syndrome after Stem-Cell Transplantation. N Engl J Med. 2017 Feb 9. 376 (6):536-547. [Medline]. [Full Text].
Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19. 127 (20):2391-405. [Medline]. [Full Text].
[Guideline] Fenaux P, Haase D, Santini V, Sanz GF, Platzbecker U, Mey U, et al. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021 Feb. 32 (2):142-156. [Medline]. [Full Text].
[Guideline] Malcovati L, Hellström-Lindberg E, Bowen D, et al. Diagnosis and treatment of primary myelodysplastic syndromes in adults: recommendations from the European LeukemiaNet. Blood. 2013 Oct 24. 122 (17):2943-64. [Medline]. [Full Text].
Malcovati L, Porta MG, Pascutto C, Invernizzi R, Boni M, Travaglino E, et al. Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol. 2005 Oct 20. 23 (30):7594-603. [Medline]. [Full Text].
Malcovati L, Della Porta MG, Strupp C, Ambaglio I, Kuendgen A, Nachtkamp K, et al. Impact of the degree of anemia on the outcome of patients with myelodysplastic syndrome and its integration into the WHO classification-based Prognostic Scoring System (WPSS). Haematologica. 2011 Oct. 96 (10):1433-40. [Medline]. [Full Text].
Garcia-Manero G, Shan J, Faderl S, Cortes J, Ravandi F, Borthakur G, et al. A prognostic score for patients with lower risk myelodysplastic syndrome. Leukemia. 2008 Mar. 22 (3):538-43. [Medline]. [Full Text].

Media Gallery

Blood film (1000× magnification) demonstrating a vacuolated blast in a refractory anemia with excess of blasts in transformation. Courtesy of U. Woermann, MD, Division of Instructional Media, Institute for Medical Education, University of Bern, Switzerland.
This bone marrow film (400× magnification) demonstrates an almost complete replacement of normal hematopoiesis by blasts in a refractory anemia with an excess of blasts in transformation. Note the signs of abnormal maturation such as vacuolation, double nucleus, and macrocytosis. Courtesy of U. Woermann, MD, Division of Instructional Media, Institute for Medical Education, University of Bern, Switzerland.
Bone marrow film (1000× magnification) demonstrating ring sideroblasts in Prussian blue staining in a refractory anemia with excess of blasts in transformation. Courtesy of U. Woermann, MD, Division of Instructional Media, Institute for Medical Education, University of Bern, Switzerland.
Bone marrow film (1000× magnification) demonstrating granular and clotlike positive reaction in periodic acid-Schiff staining in a refractory anemia with excess of blasts in transformation. Courtesy of U. Woermann, MD, Division of Instructional Media, Institute for Medical Education, University of Bern, Switzerland.

of 4

Table 1. Revised International Prognostic Scoring System risk groups and prognosis ^[19]

Risk Group	Time to Development of AML (y)	Median Survival (y)
Very low	NR	8.8
Low	10.8	5.3
Intermediate	3.2	3.0
High	1.4	1.6
Very High	0.7	0.8
AML – Acute myelogenous leukemia

Table 2 Cytogenetic abnormalities assigned an IPSS-R value for scoring

Cytogenetic prognostic subgroups	Cytogenetic abnormalities
Very good	-Y, del(11q)
Good	Normal, del(5q), del(12p), del(20q), double including del(5q)
Intermediate	Del(7q), +8, +19, t(17q), any other single or double independent clones
Poor	-7, inv(3)/t(3q)/del(3q), double including -7,/del(7q), complex: 3 abnormalities
Very poor	Complex: >3 abnormalities

Table 3.Calculation of IPSS-R score

		Points Assigned
		0	0.5	1	1.5	2	3	4
Variable	Cytogenetic subgroup	Very Good		Good		Intermediate	Poor	Very Poor
	Bone marrow blasts (%)	≤2		>2- < 5		5-10	>10
	Hemoglobin (g/dL)	≥10		8-9.9	< 8
	Platelet count (x 10⁹/L)	≥100	50-99.9	< 50
	Absolute neutrophil count (x 10⁹/L)	≥0.8	< 0.8

Table 4. IPSS-R prognostic risk scores and categories

Risk Score	Risk Category
≤1.5	Very Low
>1.5-3	Low
>3-4.5	Intermediate
>4.5-6	High
>6	Very High

Table 5. Clinical outcome by IPSS-R risk category

		IPSS-R Risk Category
		Very Low	Low	Intermediate	High	Very High
Clinical Outcome	Median survival (years)	8.8	5.3	3.0	1.6	0.8
Clinical Outcome	Median time to 25% acute myelogenous leukemia evolution (years)	NR	10.8	3.2	1.4	0.7

Table 6. Categories of FAB classification versus WHO classification for myelodysplastic syndrome (MDS)

FAB Classification	WHO–2004 Classification	WHO–2008 Classification
RA	RA RCMD 5q-	RCUD RCMD 5q-
RARS	RARS RCMD-RS	RARS RCMD-RS RARS-T
RAEB	RAEB-1 RAEB-2	RAEB-1 RAEB-2
CMML	CMML-1 CMML-2	CMML-1 CMML-2
RAEB-T	AML	AML

Table. 2008 and 2016 World Health Organization (WHO) classifications of myelodysplastic syndromes

2008 WHO Classification^[17]	2016 WHO Classification^[48]
Refractory cytopenia with unilineage dysplasia (RCUD) encompassing refractory anemia (RA), refractory neutropenia (RN), and refractory thrombocytopenia (RT)	MDS with single-lineage dysplasia (MDS-SLD)
Refractory cytopenia with multilineage dysplasia (RCMD)	MDS with multilineage dysplasia (MDS-MLD)
Refractory anemia with ringed sideroblasts (RARS)	MDS with ring sideroblasts (MDS-RS) MDS-RS with single lineage dysplasia (MDS-RS-SLD) MDS-RS with multilineage dysplasia (MDS-RS-MLD)
Myelodysplastic syndrome associated with isolated del(5q)	MDS with isolated del(5q)
	MDS with excess blasts (MDS-EB)
Refractory anemia with excess blasts–1 (RAEB-1)	MDS-EB-1
Refractory anemia with excess blasts–2 (RAEB-2)	MDS-EB-2
Myelodysplastic syndrome, unclassified (MDS-U)	MDS, unclassifiable (MDS-U) with 1% blood blasts with single lineage dysplasia and pancytopenia based on defining cytogenetic abnormality
Refractory cytopenia of childhood	Refractory cytopenia of childhood

Back to List

Myelodysplastic Syndrome (MDS) Questions & Answers

References

Tables

Contributor Information and Disclosures

What would you like to print?